| 发明名称 |
Recombinant adeno-associated virus delivery of exon 2-targeted U7snRNA polynucleotide constructs |
| 摘要 |
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy. |
| 申请公布号 |
AU2014253730(A1) |
申请公布日期 |
2015.11.12 |
| 申请号 |
AU20140253730 |
申请日期 |
2014.04.18 |
| 申请人 |
RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL |
发明人 |
FLANIGAN, KEVIN;VULIN-CHAFFIOL, ADELINE;WEIN, NICOLAS |
| 分类号 |
C07K14/47;A61K48/00;C12N15/113;C12N15/86 |
主分类号 |
C07K14/47 |
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